\n <\/p>\n
Globes of folinic acid, also called leucovorin, which Trump team officials have promoted as an intervention for autism.<\/span>Credit: Alfred Pasieka\/Science Photo Library<\/span><\/p>\n According to Martin Makary, head of the US Food and Drug Administration, the drug leucovorin will help \u201chundreds of thousands of kids\u201d with autism. But a day after Makary praised leucovorin\u2019s powers at a White House event, some specialists are warning that the science to warrant Makary\u2019s enthusiasm is far from solid.<\/p>\n Those researchers say that the drug\u2019s efficacy has not been established, that scientists don\u2019t know how much of the drug to give or how people should take it, and that safety data in children are lacking. According to the FDA\u2019s current plans, leucovorin will be available to only a minority of autistic people.<\/p>\n All of this has led to widespread confusion, say clinicians, who also worry about the expectations created by Makary and other officials in the administration of US president Donald Trump.<\/p>\n \u201cI\u2019ve heard from a lot of families,\u201d says psychologist Catherine Lord at the University of California, Los Angeles. \u201cThe major thing they say is, \u2018What is this? What do we do?\u2019\u201d<\/p>\n \u201cI don\u2019t want to get everyone\u2019s hopes up that this is a magic cure,\u201d says Rebecca Schmidt, a molecular epidemiologist at the University of California, Davis. \u201cIt\u2019s not for everybody.\u201d<\/p>\n At an announcement on 22 September, Makary announced the upcoming approval of leucovorin, a form of the vitamin folate, by saying it would \u201copen the door to the first FDA-recognized treatment pathway for autism\u201d. People with low levels of folate in the protective fluid surrounding the brain and spine can sometimes exhibit traits associated with autism, including challenges in social communication. This condition, called cerebral folate deficiency, could be due to rogue antibodies that attack the body\u2019s own proteins \u2013 in this case, proteins that ensure import of folate into the brain.<\/p>\n Trump links autism and Tylenol: is there any truth to it?<\/p>\n<\/p>\n There have been clinical trials of leucovorin, also called folinic acid, in autism, but the studies to date have been small. For example, one recent clinical trial enrolled about 80 children aged 2 to 10, and provided folinic acid supplements to about half of the participants1. Neither the participants nor their physicians knew who received the supplement and who received a placebo. Participants who received the supplement reported greater improvements in social interactions and language skills than those who received the placebo.<\/p>\n After the trial was published, some researchers subsequently raised concerns that the assessment of those improvements was subjective, and that the study was too small to detect subtle differences in response2.<\/p>\n But size is not the only thing that matters in clinical trials, says Dan Rossignol, a family physician in Aliso Viejo, California, who has studied the data on leucovorin and sometimes prescribes it to autistic children. The effect of leucovorin in the trials has been large enough to be apparent even with small numbers of participants, he says. Specifically, Rossignol points to an early leucovorin clinical study, in which only 48 children participated but some experienced marked improvements in a standardized assessment of speech3.<\/p>\n \u201cBut it would be great if more studies were done with more kids,\u201d he says. \u201cThen we could tease out which kids respond better.\u201d Typically, studies submitted for FDA approval of a drug for autism might have data from hundreds of children, he says, but it has been difficult to raise the money for bigger trials. Rossignol says that he and a colleague have been in discussions with US President Donald Trump\u2019s administration to make the case for leucovorin.<\/p>\n On Monday, the Trump administration said the US National Institutes of Health plans to monitor the effects of the FDA\u2019s anticipated approval and to study possible broader benefits of leucovorin in autistic people. No details have been released as to how such studies will be designed.<\/p>\n Regardless of the therapy\u2019s efficacy, it will not be a panacea. The FDA\u2019s proposed approval, which has not yet been finalized, would apply only to people with low levels of folate in the fluid surrounding the brain and spinal cord. That represents about 7\u201430% of autistic people, depending on how folate levels are measured, says Alycia Halladay, chief science officer of the Autism Science Foundation in New York City. \u201cFamilies are in the Facebook groups saying, \u2018We can get a prescription now\u2019,\u201d she says. \u201cAnd that\u2019s not what\u2019s going to happen.\u201d<\/p>\n Autism is complex and thought to be caused by a wide range of factors. No single therapy is likely to apply to all autistic people. \u201cIf anyone tells you they have found a magic bullet cure for autism, doubt them,\u201d says Halladay. \u201cThere is not going to be one cause and one singular treatment.\u201d<\/p>\n Autism is on the rise: what\u2019s really behind the increase?<\/p>\n<\/p>\n The lack of data from large trials means that doctors will have little to go on when determining what dose to prescribe, or how long children should take leucovorin before deciding if it is or is not working, says Halladay. \u201cWe hope there\u2019s eventually going to be guidance,\u201d she says. Rossignol says that most of the clinical studies of leucovorin in autistic children have used the same dose, which could provide a starting point for physicians.<\/p>\n","protected":false},"excerpt":{"rendered":" Globes of folinic acid, also called leucovorin, which Trump team officials have promoted as an intervention for autism.Credit: Alfred Pasieka\/Science Photo Library According to Martin Makary, head of the US Food and Drug Administration, the drug leucovorin will help \u201chundreds of thousands of kids\u201d with autism. But a day after Makary praised leucovorin\u2019s powers at<\/p>\n","protected":false},"author":1,"featured_media":23421,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[58],"tags":[5814,1348,252,1905,81,14172,514],"class_list":{"0":"post-23420","1":"post","2":"type-post","3":"status-publish","4":"format-standard","5":"has-post-thumbnail","7":"category-science","8":"tag-autism","9":"tag-backs","10":"tag-drug","11":"tag-team","12":"tag-trump","13":"tag-unproven","14":"tag-work"},"_links":{"self":[{"href":"https:\/\/naijaglobalnews.org\/index.php?rest_route=\/wp\/v2\/posts\/23420","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/naijaglobalnews.org\/index.php?rest_route=\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/naijaglobalnews.org\/index.php?rest_route=\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/naijaglobalnews.org\/index.php?rest_route=\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/naijaglobalnews.org\/index.php?rest_route=%2Fwp%2Fv2%2Fcomments&post=23420"}],"version-history":[{"count":0,"href":"https:\/\/naijaglobalnews.org\/index.php?rest_route=\/wp\/v2\/posts\/23420\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/naijaglobalnews.org\/index.php?rest_route=\/wp\/v2\/media\/23421"}],"wp:attachment":[{"href":"https:\/\/naijaglobalnews.org\/index.php?rest_route=%2Fwp%2Fv2%2Fmedia&parent=23420"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/naijaglobalnews.org\/index.php?rest_route=%2Fwp%2Fv2%2Fcategories&post=23420"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/naijaglobalnews.org\/index.php?rest_route=%2Fwp%2Fv2%2Ftags&post=23420"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}Vitamin in the spotlight<\/h2>\n
Call for bigger trials <\/h2>\n
Not a magic bullet<\/h2>\n